Izcargo, a biological product that crosses the blood-brain barrier, was approved for the first time on the market

A few days ago, JCR Pharmaceuticals announced that the Ministry of Health, Labour and Welfare (MHLW) of Japan has approved Izcargo (pabinafusp alfa) for the treatment of mucopolysaccharidosis type II (MPS II, also known as Hunter syndrome).

Due to genetic mutations in MPS II patients, the key metabolic protease is defective or missing, which causes the accumulation of toxic metabolites in the lysosome.

Izcargo is a recombinant fusion protein that fuses IDS protease with an antibody targeting human transferrin receptor, and uses transferrin receptor-mediated transcytosis to help cross the blood-brain barrier.

The approval of Izcargo by the Japanese Ministry of Health, Labour and Welfare is based on comprehensive evidence from preclinical and clinical studies.

Note: The original text has been deleted

Reference materials:

[1] JCR Pharmaceuticals Announces Approval of IZCARGO® (Pabinafusp Alfa) for Treatment of MPS II (Hunter Syndrome) in Japan.

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